The Impact of Indication Extensions on Pharmaceutical Prices

Genane, C.; Marinoni, G.; Andò, Giuseppe; Reinaud, F.

doi:10.1016/j.jval.2013.08.759

Cited by 2 publications

(1 citation statement)

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“…Indeed, previous research showed that orphan drug prices are determined based on the prevalence of the first indication [ 36 , 45 ]. Launch prices for the first indication are unlikely to be reviewed following approval in other indications [ 46 ]. We calculated that orphan drugs with multiple indications have a significantly larger potential market size, although it is essential to bear in mind that no correct estimates of prevalence exist and not all patients are diagnosed and/or treated [ 47 ].…”

Section: Discussionmentioning

confidence: 99%

Shining a light in the black box of orphan drug pricing

Picavet

Morel

Cassiman

et al. 2014

Orphanet Journal of Rare Diseases

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BackgroundThe pricing mechanism of orphan drugs appears arbitrary and has been referred to as a “black box”. Therefore, the aim of this study is to investigate how drug- and disease-specific variables relate to orphan drug prices. Additionally, we aim to explore if certain country-specific pricing and reimbursement policies affect the price level of orphan drugs.MethodsAnnual treatment costs per indication per patient were calculated for 59 orphan drugs with a publicly available price in Belgium, the Netherlands, Czech Republic, France, Italy and the United Kingdom. A multiple linear regression model was built with 14 drug- and disease-specific variables. A Mann-Whitney U test was used to explore whether there is a correlation between annual treatment costs of orphan drugs across countries with different pricing and reimbursement policies.ResultsRepurposed orphan drugs, orally administered orphan drugs or orphan drugs for which an alternative treatment is available are associated with lower annual treatment costs. Orphan drugs with multiple orphan indications, for chronic treatments or for which an improvement in overall survival or quality-of-life has been demonstrated, are associated with higher annual treatment costs. No association was found between annual treatments cost of orphan drugs across countries and the different pricing and reimbursement systems.ConclusionsThis study has shown that prices of orphan drugs are influenced by factors such as the availability of an alternative drug treatment, repurposing, etc. Current debate about the affordability of orphan drugs highlights the need for more transparency in orphan drug price setting.

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Section: Discussionmentioning

confidence: 99%

Shining a light in the black box of orphan drug pricing

Picavet

Morel

Cassiman

et al. 2014

Orphanet Journal of Rare Diseases

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Gene Therapies: The Challenge of sUper-High-Cost Treatments and How to Pay for them

Carr

Bradshaw

2016

Regen. Med.

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Gene therapies have the potential to cure rare conditions that often have no current efficacious treatments with a one-time treatment episode, relieving substantial unmet need and having profound positive impact on patients' lives. However, with the first gene therapy now licensed and priced at around US$1 million per patient, cost and uncertain funding mechanisms present a potential barrier to patient access. In this article, we discuss the unique challenges presented by gene therapies, particularly concerning the uncertainty inherent in their clinical evidence package at launch and their affordability within strained healthcare budgets. We present several payment models that would allow for sustainable reimbursement of these innovative technologies and make recommendations pertinent both to those developing gene therapies and to those paying for them.

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The Impact of Indication Extensions on Pharmaceutical Prices

Cited by 2 publications

References 0 publications

Shining a light in the black box of orphan drug pricing

Shining a light in the black box of orphan drug pricing

Gene Therapies: The Challenge of sUper-High-Cost Treatments and How to Pay for them

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